Request for Information (RFI): Input on Administration of the NIH-Industry Pilot Program Discovering New Therapeutic Uses for Existing Molecules

Notice Number:NOT-RM-13-021

Key DatesRelease Date: June 14, 2013Response Date: July 18, 2013

Issued by
National Institutes of Health (NIH)
Office of Strategic Coordination (Common Fund)

Purpose

The goal of this Request for Information (RFI) is to collect feedback from the biomedical research community, pharmaceutical companies, and other members of the public about the “NIH-Industry Pilot Program: Discovering New Therapeutic Uses for Existing Molecules” initiative and the application process. NCATS is interested in feedback from researchers and institutions that submitted an application, considered submitting an application but did not, and/or would be interested in submitting an application in the future. NCATS is also interested in feedback from existing and potential pharmaceutical partners.

Background

In June 2012, NCATS issued the “Discovering New Therapeutic Uses for Existing Molecules” Funding Opportunity Announcements (FOAs) (PAR-12-203, RFA-TR-12-004, RFA-TR-12-005), inviting U.S. investigators to submit applications to test new therapeutic uses for 58 proprietary compounds and biologics (Agents) provided by pharmaceutical partners. With the intent of accelerating the process of developing new therapies for patients, this pilot program funds pre-clinical through phase 2a clinical trials that bridge the identification of new therapeutic uses for Agents with known pharmacologic mechanisms of action to a stage that pharmaceutical partners can further develop (phase 3 clinical trials). Through this pilot program ideas for new uses for the multiple Agents were requested from the collective intelligence of the research community in all disease areas. This is one way that NCATS is working to facilitate win-win collaborations that enable components of the research ecosystem to work together more effectively.

Prior to issuance of the “Discovering New Therapeutic Uses for Existing Molecules” FOAs, memorandums of understanding (MOU) were established between the NIH and each of eight pharmaceutical companies that were participating in this pilot program. Pre-applications (X02) for new uses for the Agents were solicited from the research community through PAR-12-203. In this pre-application announcement limited non-confidential information about the available Agents (e.g., mechanism of action, route of administration, and any limitations in use based on safety and tolerability studies) were available on the NCATS website so investigators could decide whether an Agent might be appropriate for their disease of interest. In addition, investigators were asked to work with their institutions to assess feasibility of executing template agreements (see description below). The pre-application (X02) receipt date was two months after the FOA was issued.

NIH peer review assessed the scientific merit of the X02 pre-applications, and the most meritorious applicants had an opportunity to submit full applications via one of two possible funding mechanisms, a UH2/UH3 (RFA-TR-12-004) or a UH3 (RFA-TR-12-005). A UH2/UH3 award provides up to three years of support for studies that aim to establish evidence for a new therapeutic use for an Agent. A UH3 award provides two years of support for clinical trials that aim to establish proof of concept for the use of an Agent in a specified disease population.

To streamline the collaboration initiation between applicants at academic medical centers and the pharmaceutical partners and to provide a roadmap for handling of confidentiality and intellectual property used or developed through the program, template agreements [Confidential Disclosure Agreement (CDA) and Collaborative Research Agreement (CRA)], were made available to potential applicants on the NCATS website. Applicants with an opportunity to submit a UH2/UH3 or UH3 application were placed in contact with the pharmaceutical company partner to execute a Confidential Disclosure Agreement (CDA). The CDA established a way to exchange confidential information needed by the applicants in advance of submitting a UH2/UH3 or UH3 application. A CRA/documentation of access to the Agent and associated data needed for conducting the proposed studies was required when the UH2/UH3 or UH3 application was submitted.

Information Requested

NCATS is interested in obtaining comments about the pre-application (X02) process and full application process (UH2/UH3 or UH3). Comments are invited for but not limited to the following:

1. Reasons why your institution would be interested in submitting a pre-application but not participate.
2. The amount of public information that was posted for the available Agents in terms of the utility of the information for proposing studies for new therapeutic uses.
3. Inclusion or absence of target classes represented by the 58 available Agents.
4. NIH used a peer review process to determine the scientific merit of the X02 pre-applications. Pharmaceutical partners were not involved in the review process. Comments can include the perceived advantages/disadvantages of this approach, how pharmaceutical partners might participate in the future and considerations that should be given if NIH allows the pharmaceutical partners to review the pre-applications.
5. If you were an X02 applicant and put in contact with a pharmaceutical company by NCATS to discuss your application, feel free to comment on the feedback you received from the company.
6. Any institutional challenges with getting CDAs and CRAs in place and the perceived value of the template agreements.
7. Your level of interest in responding to an FOA for Discovering New Therapeutic Uses for Existing Molecules in the future.
8. Your level of interest in being a pharmaceutical partner in the future.
9. If you have additional comments or feedback that was not addressed previously, include it here.

You will see an electronic confirmation acknowledging receipt of your response, but will not receive individualized feedback. Submitted information will not be considered confidential, although each submission will be stored using a login and a password. No proprietary, classified, confidential, or sensitive information should be included in your response. NCATS staff may contact any responder for the sole purpose of enhancing NCATS’ understanding of the RFI submission.

NCATS will use the submitted information at its discretion. This RFI notice should not be interpreted as a solicitation or as an obligation on the part of the Federal Government, NIH, or NCATS. No monetary awards will be made to pay for preparation of any information submitted or for the Government’s use of such information. No basis for claims against the U.S. Government shall arise as a result of a response to this request for information or from the Government’s use of such information.

General Information
Note: All of the following fields are optional. Proprietary, classified, confidential, or sensitive information should not be included in your response.

1. Please identify the nature of your interest in the area [e.g., whether you are a biomedical or clinical researcher, a pharmaceutical company interested in participating in this initiative, a member of an advocacy or community group, or other (please indicate)].
2. Please indicate the name of the organization, if any.
3. Please indicate your main area of research interest.
4. Please indicate your name.
5. Please indicate your email address.